Using a new experimental technique called CRISPR base editor, a team led by University of Wisconsin researchers was able to repair a gene mutation that causes childhood blindness known as Leber congenital amaurosis (LCA). The scientists showed that their approach worked in lab-grown cells derived from patients with the currently untreatable inherited disease and in a mouse model.
The method involves delivering CRISPR base editor to retinal cells using silica nanocapsules, which are designed to target specific cell types for therapy. The goal is to create a package that can carry these editors to the eye, allowing them to introduce changes in the DNA sequence of affected cells.
The researchers believe their technique has several advantages over other methods, including its ability to correct specific errors in the DNA sequence and avoid unpredictable immune system responses. By using CRISPR base editor, they were able to overcome challenges through a collaborative team science approach that involved scientists from UW-Madison, Harvard, and MIT.
The study was published in the Journal of Clinical Investigation and showed promise for treating not only LCA but also another form of childhood blindness called Best disease (also known as Best vitelliform macular dystrophy). The researchers are hopeful that their work will lead to new treatments for these conditions, which cause extreme farsightedness, sensitivity to light, involuntary eye movements, and loss of central vision.